Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy: A multicentric longitudinal study using the Performance of Upper Limb test

Marika Pane, Lavinia Fanelli, Elena Stacy Mazzone, Giorgia Olivieri, Adele D'Amico, Sonia Messina, Marianna Scutifero, Roberta Battini, Roberta Petillo, Silvia Frosini, Serena Sivo, Gian Luca Vita, Claudio Bruno, Tiziana Mongini, Elena Pegoraro, Roberto De Sanctis, Alice Gardani, Angela Berardinelli, Valentina Lanzillotta, Adelina CarlesiEmanuela Viggiano, Filippo Cavallaro, Maria Sframeli, Luca Bello, Andrea Barp, Flaviana Bianco, Serena Bonfiglio, Enrica Rolle, Concetta Palermo, Grazia D'Angelo, Antonella Pini, Elena Iotti, Ksenija Gorni, Giovanni Baranello, Enrico Bertini, Luisa Politano, Maria Pia Sormani, Eugenio Mercuri

Research output: Contribution to journalArticlepeer-review

Abstract

The aim of this study was to establish the possible effect of glucocorticoid treatment on upper limb function in a cohort of 91 non-ambulant DMD boys and adults of age between 11 and 26 years.All 91 were assessed using the Performance of Upper Limb test. Forty-eight were still on glucocorticoid after loss of ambulation, 25 stopped steroids at the time they lost ambulation and 18 were GC naïve or had steroids while ambulant for less than a year.At baseline the total scores ranged between 0 and 74 (mean 41.20). The mean total scores were 47.92 in the glucocorticoid group, 36 in those who stopped at loss of ambulation and 30.5 in the naïve group (p <0.001).The 12-month changes ranged between -20 and 4 (mean -4.4). The mean changes were -3.79 in the glucocorticoid group, -5.52 in those who stopped at loss of ambulation and -4.44 in the naïve group. This was more obvious in the patients between 12 and 18 years and at shoulder and elbow levels.Our findings suggest that continuing glucocorticoids throughout teenage years and adulthood after loss of ambulation appears to have a beneficial effect on upper limb function.

Original languageEnglish
Pages (from-to)749-753
Number of pages5
JournalNeuromuscular Disorders
Volume25
Issue number10
DOIs
Publication statusPublished - Oct 1 2015

Keywords

  • Duchenne muscular dystrophy
  • Glucocorticoids
  • Non ambulant
  • PUL
  • Upper limb

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health
  • Genetics(clinical)
  • Neurology

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